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Amylyx Pharmaceuticals halts Amx0035's Orion program for PSP, shifting focus to Avexitide and other promising treatments for neurodegenerative diseases.
Amx0035 failed to show differences compared to placebo at the end of week 24 prompting the end of the trial.
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Amylyx Pharmaceuticals announced it has discontinued the Orion program of Amx0035 in adults suffering from progressive supranuclear palsy (PSP). Amx0035’s Orion program was discontinued due to Amx0035 not showing any difference compared to placebo on both primary and secondary outcomes by week 24.
Based on these results, Amylyx announced it will not be moving forward with Phase III of the program, but recorded safety data consistent with safety data from previous studies showing Amx0035 continues to be well-tolerated.
Camille L. Bedrosian, MD, chief medical officer at Amylyx, touched on the decision to discontinue the Orion program, saying, “We set a high bar for AMX0035 in PSP and made a commitment to base our decision-making on the totality of the data and the potential for clinically meaningful outcomes for those living with PSP. While we are disappointed in these results, we believe these data will inform the PSP trial literature as well as deepen scientific understanding of this devastating disease. We extend our gratitude to the participants, their families and care partners, the Orion sites, and the entire PSP community for their collaboration on this study,”1
AMX0035 is an orally administered, fixed-dose combination of sodium phenylbutyrate and taurursodiol (Turso, also known as ursodoxicoltaurine outside of the U.S.). Amx0035 is designed to mitigate neurodegeneration through targeting endoplasmic reticulum stress and mitochondrial dysfunction, two connected central pathways that lead to cell death and neurodegeneration.1 Amylyx believes the combination of phenylbutyrate and taurursodiol’s complementary mechanisms of action will synergistically target abnormal cell death, in turn preventing neurodegeneration better than other treatments targeted at a singular action.
Progressive supranuclear palsy (PSP) is a sporadic, rare, and fatal neurodegenerative disorder affecting movement, gait, balance, eye movements, swallowing, and speech.1 Affecting an estimated seven in 100,000 people worldwide, patients diagnosed with PSP have a life expectancy ranging from six to eight years, following their initial diagnosis. PSP is mainly diagnosed during a patient’s late-middle-ages and rapidly progresses over time.
Joshua Cohen and Justin Klee, Co-CEOs of Amylyx, discussed Amylyx’s shift in focus to Avexititde following Amx0035’s discontinued Phase II trial, saying, “Amylyx remains committed to advancing potential new treatments for communities with high unmet needs. Our highest priority and focus remain on the pivotal Phase 3 LUCIDITY trial of avexitide, with enrollment expected to complete in 2025 and topline data anticipated in the first half of 2026. We are also continuing development of AMX0035 in Wolfram syndrome and progressing AMX0114 in ALS, with early cohort data from the Phase 1 LUMINA trial expected in 2025.”1
Amylyx’s avexitide is an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist, evaluated in five Phase I and Phase II clinical trials for post-bariatric hypoglycemia (PBH). 1 Avexitide has also been studied in congenital hyperinsulinism (HI), along with being granted FDA Breakthrough Therapy Designation for both indications of rare pediatric disease designation in congenital HI, and orphan drug designation for the treatment of hyperinsulinemic hypoglycemia. Avexitide is designed to bind to the GLP-1 receptor on pancreatic islet beta cells and inhibit the effect of GLP-1 to mitigate hypoglycemia by decreasing insulin secretion and stabilizing blood glucose levels.
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